Pediatric Neurology Leukodystrophy Reference Center, Pediatric Neurology Department, Université Paris-Saclay, Hôpital Bicêtre, France


Dr. Caroline Sevin is a pediatric neurologist at Kremlin Bicêtre Hospital. She leads the French reference centre for leukodystrophies in children at Kremlin Bicêtre Hospital, France.

‪She has long-standing interests ‬ in neurodegenerative metabolic diseases, including lysosomal, peroxysomal and mitochondrial diseases. She has a strong background in leukodystrophies, particularly ALD, MLD and Krabbe disease. CS is PI or co-PI of several clinical trials, mostly in ALD and MLD, using innovative therapies (gene therapy, intrathecal enzyme replacement therapy, small molecules). Dr Caroline Sevin is a member of the ERN-RND and MLDi consortium.

She’s a member of several DSMBs for gene therapy trials and a member of the ASGCT, SSIEM, EAN, and EPNS. Her research focuses on developing in vivo/ex vivo gene therapy approaches for leukodystrophies.‬